ALS News Today

ALS gene therapy targeting TDP-43 protein put on FDA fast track

The FDA granted fast track status to VectorY’s VTx-002, an antibody gene therapy targeting TDP-43 protein aggregates in ALS.
WOOD-TV

Locanabio Presents Preclinical Data from its Vectorized snRNA Exon Skipping Program for DMD and Cas13d Multi-targeting Program for C9orf72 ALS at the American Society of Gene ...

LBIO-115 demonstrates robust exon 51 skipping resulting in high levels of dystrophin restoration in patient-derived muscle cells and an in vivo DMD disease model Cas13d Multi-targeting efficiently ...

Belief Biomed Congratulates Partner Askbio On IND Acceptance By FDA For Investigational Gene Therapy For Late-Onset Pompe Disease

Belief Biomed Congratulates Partner Askbio On IND Acceptance By FDA For Investigational Gene Therapy For Late-Onset Pompe Disease. SHANGHAI, Jan. 14, 2026 /PRNewswire/ -- AskBio Inc. (AskBio), a gene ...
GEN

Scalable CRISPR-Based Gene Editing Improves Tomato Crop Traits

In a study published in Nature Communications titled “Construction of multi-targeted CRISPR libraries in tomato to overcome functional redundancy at genome-scale level,” researchers from Tel Aviv ...